Treatment series focused on MPN-AP or MPN-BP
| Treatment approach . | Patient population . | Comments . | Survival . | Reference . |
|---|---|---|---|---|
| IC ± allogeneic HCT | MPN-BP (n = 91) | 24 (26%) received IC; 41% of IC group had return to cMPN | OS, 2.6 mo; 3.9 mo in IC group; 1 patient underwent allogeneic HCT | 8 |
| MPN-BP (n = 74) | 41 (55%) received IC; CR/CRi, 46% | OS, 5 mo; OS, 6 mo and PFS, 5 mo with IC; 8 patients underwent early SCT; 73% of these were alive at median of 31 mo | 9 | |
| MPN-BP (n = 75) | 39 (51%) treated with curative intent and received IC and/or underwent allogeneic HCT | OS, 6.6 mo; median OS of curative intent group, 9.4 mo; 17 (23%) underwent allogeneic HCT; median OS, 47 mo for allogeneic HCT | 50 | |
| MPN-BP (n = 248) | 66 of 122 patients assessable for response received IC; 26 received HMA | OS, 3 mo; 1-y survival before y 2000, 5%; it was 20% in ≥2010; CR, 35% with IC; CR <5% with HMA | 10 | |
| HMAs | MPN-BP (n = 26); MPN-AP (n = 28) | ORR, 52% (CR, 24%) with azacitidine therapy; median response duration, 9 mo; recurrence of cMPN in 39% responders | OS, 11 mo | 67 |
| MPN-BP (n = 19) | ORR, 47% (CR, 26%) with azacitidine therapy | OS, 9.9 mo | 64 | |
| MPN-BP (n = 21); MPN-AP (n = 13); PMF chronic phase (n = 11) | ORR in MPN-BP, 29% with decitabine therapy; median response duration, 7 mo | OS, 6.9 mo in MPN-BP; OS, 9.7 mo in MPN-AP | 65 | |
| MPN-BP (n = 11) | 6 patients received decitabine; 5 patients underwent allogeneic HCT | 67% in decitabine group alive at 9 mo; 53% in transplantation group alive at 20 mo | 66 | |
| JAK inhibition | R/R AML (n = 38), including MPN-BP (n = 18) | 3 of 18 patients with MPN-BP treated with ruxolitinib at dose of 25 mg twice daily achieved CR/CRi | NR | 77 |
| R/R acute leukemia (n = 28), including 7 with antecedent Ph− MPN | 1 patient with AML (prior MDS) achieved CRp; no objective response in MPN-BP cohort; ruxolitinib dosed between 50 and 200 mg twice daily | NR | 78 | |
| JAK inhibition + HMA | MPN-AP/MPN-BP (n = 21) | Phase 1 study of ruxolitinib + decitabine; 7 of 21 responded | OS, 10.4 mo | 80 |
| MPN-BP (n = 10) | Phase 1/2 study of ruxolitinib + decitabine | NR | 79 | |
| Allogeneic HCT | MPN-BP (n = 43); MDS/MPN-BP (n = 17) | 60 underwent allogeneic HCT; 3-y TRM, 22%; 3-y CIR, 68% | OS, 18% at 3 y; 3-y LFS, 18% for allogeneic HCT in CR; 3-y LFS, 3% for allogeneic HCT in advanced disease | 47 |
| MPN-BP (n = 13) | 8 underwent allogeneic HCT; 5 of 8 were in CR or cMPN at time of allogeneic HCT | At median follow-up of 20 mo, 6 patients were alive in CR post–allogeneic HCT | 48 | |
| MF chronic phase (n = 41); MF-BP (n = 14) | Of 14 MF-BP patients who underwent allogeneic HCT, 7 survived; median follow-up, 31 mo | OS, 49% at 2 y in MF-BP cohort; 3 of 6 patients in remission and 4 of 8 patients with relapsed disease at allogeneic HCT were long-term survivors | 49 | |
| MF-BP (n = 46) | 1-y TRM, 28%, CIR at 3 y, 47%; only 8 of 46 were in CR pre–allogeneic HCT | 3-y PFS, 26%; OS, 33%; CR pre–allogeneic HCT was significantly predictive of OS and PFS | 46 |
| Treatment approach . | Patient population . | Comments . | Survival . | Reference . |
|---|---|---|---|---|
| IC ± allogeneic HCT | MPN-BP (n = 91) | 24 (26%) received IC; 41% of IC group had return to cMPN | OS, 2.6 mo; 3.9 mo in IC group; 1 patient underwent allogeneic HCT | 8 |
| MPN-BP (n = 74) | 41 (55%) received IC; CR/CRi, 46% | OS, 5 mo; OS, 6 mo and PFS, 5 mo with IC; 8 patients underwent early SCT; 73% of these were alive at median of 31 mo | 9 | |
| MPN-BP (n = 75) | 39 (51%) treated with curative intent and received IC and/or underwent allogeneic HCT | OS, 6.6 mo; median OS of curative intent group, 9.4 mo; 17 (23%) underwent allogeneic HCT; median OS, 47 mo for allogeneic HCT | 50 | |
| MPN-BP (n = 248) | 66 of 122 patients assessable for response received IC; 26 received HMA | OS, 3 mo; 1-y survival before y 2000, 5%; it was 20% in ≥2010; CR, 35% with IC; CR <5% with HMA | 10 | |
| HMAs | MPN-BP (n = 26); MPN-AP (n = 28) | ORR, 52% (CR, 24%) with azacitidine therapy; median response duration, 9 mo; recurrence of cMPN in 39% responders | OS, 11 mo | 67 |
| MPN-BP (n = 19) | ORR, 47% (CR, 26%) with azacitidine therapy | OS, 9.9 mo | 64 | |
| MPN-BP (n = 21); MPN-AP (n = 13); PMF chronic phase (n = 11) | ORR in MPN-BP, 29% with decitabine therapy; median response duration, 7 mo | OS, 6.9 mo in MPN-BP; OS, 9.7 mo in MPN-AP | 65 | |
| MPN-BP (n = 11) | 6 patients received decitabine; 5 patients underwent allogeneic HCT | 67% in decitabine group alive at 9 mo; 53% in transplantation group alive at 20 mo | 66 | |
| JAK inhibition | R/R AML (n = 38), including MPN-BP (n = 18) | 3 of 18 patients with MPN-BP treated with ruxolitinib at dose of 25 mg twice daily achieved CR/CRi | NR | 77 |
| R/R acute leukemia (n = 28), including 7 with antecedent Ph− MPN | 1 patient with AML (prior MDS) achieved CRp; no objective response in MPN-BP cohort; ruxolitinib dosed between 50 and 200 mg twice daily | NR | 78 | |
| JAK inhibition + HMA | MPN-AP/MPN-BP (n = 21) | Phase 1 study of ruxolitinib + decitabine; 7 of 21 responded | OS, 10.4 mo | 80 |
| MPN-BP (n = 10) | Phase 1/2 study of ruxolitinib + decitabine | NR | 79 | |
| Allogeneic HCT | MPN-BP (n = 43); MDS/MPN-BP (n = 17) | 60 underwent allogeneic HCT; 3-y TRM, 22%; 3-y CIR, 68% | OS, 18% at 3 y; 3-y LFS, 18% for allogeneic HCT in CR; 3-y LFS, 3% for allogeneic HCT in advanced disease | 47 |
| MPN-BP (n = 13) | 8 underwent allogeneic HCT; 5 of 8 were in CR or cMPN at time of allogeneic HCT | At median follow-up of 20 mo, 6 patients were alive in CR post–allogeneic HCT | 48 | |
| MF chronic phase (n = 41); MF-BP (n = 14) | Of 14 MF-BP patients who underwent allogeneic HCT, 7 survived; median follow-up, 31 mo | OS, 49% at 2 y in MF-BP cohort; 3 of 6 patients in remission and 4 of 8 patients with relapsed disease at allogeneic HCT were long-term survivors | 49 | |
| MF-BP (n = 46) | 1-y TRM, 28%, CIR at 3 y, 47%; only 8 of 46 were in CR pre–allogeneic HCT | 3-y PFS, 26%; OS, 33%; CR pre–allogeneic HCT was significantly predictive of OS and PFS | 46 |
CIR, cumulative incidence of relapse; cMPN, reversion to chronic phase MPN; CR, complete response; CRi, CR with incomplete hematologic recovery; HCT, hematopoietic cell transplantation; HMA, hypomethylating agent; IC, intensive chemotherapy; NR, not reported; ORR, overall response rate; OS, overall survival; PFS, progression-free survival; R/R, relapsed/refractory; TRM, transplantation-related mortality.